JP5706602B2 - ニーマンピック病ａ型に対する遺伝子治療 - Google Patents

ニーマンピック病ａ型に対する遺伝子治療 Download PDF

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JP5706602B2
JP5706602B2 JP2008554342A JP2008554342A JP5706602B2 JP 5706602 B2 JP5706602 B2 JP 5706602B2 JP 2008554342 A JP2008554342 A JP 2008554342A JP 2008554342 A JP2008554342 A JP 2008554342A JP 5706602 B2 JP5706602 B2 JP 5706602B2
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aav2
vector
combination according
brain
transgene
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JP2009526067A (ja
JP2009526067A5 (https=
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マルコ・エイ・パッシーニ
ロビン・ジェイ・ジーグラー
ジェイムズ・ドッジ
ラムヤ・シハブッディン
セン・エイチ・チェン
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Genzyme Corp
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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2227/10—Mammal
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2008554342A 2006-02-08 2007-02-08 ニーマンピック病ａ型に対する遺伝子治療 Active JP5706602B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US77162806P 2006-02-08 2006-02-08
US60/771,628 2006-02-08
US77236006P 2006-02-09 2006-02-09
US60/772,360 2006-02-09
PCT/US2007/003388 WO2007092563A2 (en) 2006-02-08 2007-02-08 Gene therapy for niemann-pick disease type a

Related Child Applications (1)

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JP2013092907A Division JP5881641B2 (ja) 2006-02-08 2013-04-25 ニーマンピック病ａ型に対する遺伝子治療

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JP2009526067A JP2009526067A (ja) 2009-07-16
JP2009526067A5 JP2009526067A5 (https=) 2010-04-08
JP5706602B2 true JP5706602B2 (ja) 2015-04-22

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JP2008554342A Active JP5706602B2 (ja) 2006-02-08 2007-02-08 ニーマンピック病ａ型に対する遺伝子治療
JP2013092907A Active JP5881641B2 (ja) 2006-02-08 2013-04-25 ニーマンピック病ａ型に対する遺伝子治療

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US (3) US20090117156A1 (https=)
EP (2) EP3456331B1 (https=)
JP (2) JP5706602B2 (https=)
AR (1) AR059371A1 (https=)
BR (1) BRPI0707590A2 (https=)
CY (1) CY1121380T1 (https=)
DK (1) DK1986661T3 (https=)
ES (1) ES2700048T3 (https=)
HU (1) HUE041928T2 (https=)
IL (2) IL193165B (https=)
LT (1) LT1986661T (https=)
MX (3) MX360595B (https=)
PL (1) PL1986661T3 (https=)
PT (1) PT1986661T (https=)
SI (1) SI1986661T1 (https=)
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WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis
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AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
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KR20230031329A (ko) * 2020-06-30 2023-03-07 칠드런스 내셔널 메디컬 센터 골격 근섬유 복구를 개선하기 위한 재조합 인간 산성 스핑고미엘리나제의 용도
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JP2024506860A (ja) * 2021-02-01 2024-02-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ニーマンピック病ａ型を治療するための組成物及び方法

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DK1986661T3 (da) 2019-01-02
IL193165B (en) 2018-04-30
AR059371A1 (es) 2008-03-26
JP2009526067A (ja) 2009-07-16
US20230287366A1 (en) 2023-09-14
CY1121380T1 (el) 2020-05-29
EP1986661A2 (en) 2008-11-05
JP2013166777A (ja) 2013-08-29
US20090117156A1 (en) 2009-05-07
EP3456331A1 (en) 2019-03-20
MX360595B (es) 2018-11-09
LT1986661T (lt) 2018-12-10
BRPI0707590A2 (pt) 2011-05-10
MX2018013684A (es) 2021-06-15
EP1986661A4 (en) 2012-04-25
JP5881641B2 (ja) 2016-03-09
IL258587A (en) 2018-06-28
PT1986661T (pt) 2018-12-05
PL1986661T3 (pl) 2019-02-28
WO2007092563A3 (en) 2007-11-22
US20200080066A1 (en) 2020-03-12
SI1986661T1 (sl) 2018-12-31
MX2018008355A (es) 2021-06-15
WO2007092563A2 (en) 2007-08-16
HUE041928T2 (hu) 2019-06-28
EP3456331B1 (en) 2021-05-26
ES2700048T3 (es) 2019-02-13
EP1986661B1 (en) 2018-08-29

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